Growth hormone prescribing patterns in the UK, 2013–2016

Abstract

IntroductionPrescribing of recombinant human growth hormone (rhGH) for growth failure in UK children is based on guidance from the National Institute for Health and Care Excellence. In 2013, the British Society for Paediatric Endocrinology and Diabetes initiated a national audit of newly prescribed rhGH treatment for children and adolescents. In this review, we have examined prescribing practices between 2013 and 2016.MethodsAll patients ≤16.0 years of age starting rhGH for licensed and unlicensed conditions in the UK were included. Anonymised data on indication and patient demographics were analysed.ResultsDuring the 4 years, 3757 patients from 76 of 85 (89%) centres started rhGH. For each licensed indication, proportions remained stable over this period: 56% growth hormone deficiency (GHD), 17% small for gestational age (SGA), 10% Turner syndrome, 6% Prader-Willi syndrome (PWS), 3% chronic renal insufficiency (CRI) and 2% short stature homeobox deficiency (SHOXd). However, the unlicensed category decreased from 10% (n=94) in 2013 to 5% (n=50) in 2016. The median age of patients starting rhGH was 7.6 years (range 0.1–16.0). Patients with PWS were significantly younger (median 2.2 years, range 0.2–15.1) compared with other indications (p<0.0001) and were followed by the SGA group (median 6.2 years, range 1.3–15.6, p<0.0001). Boys predominated in all groups except for PWS and SHOXd.ConclusionWe demonstrate significant engagement of prescribing centres in this audit and a decline in unlicensed prescribing by half in this 4-year period. Patients in the PWS group were younger at initiation of rhGH compared with other indications and had no male predominance unlike GHD, SGA and CRI.

Real-World Treatment Patterns and Outcomes of Growth Hormone Treatment Among Children in Israel Over the Past Decade (2004–2015)

“…The available evidence suggests that success of GH therapy depends on a number of factors including age at initiation of treatment (6), adherence (9), indication, and gender (10). Most of the current information regarding treatment patterns and long-term outcome of GH therapy is derived from pharmaceutical based registries [Kabi International Growth Study (KIGS), NordiNet R International Outcome Study, ANSWER] and several nationwide cohorts (11)(12)(13)(14), which provide limited data on the baseline characteristics at GH initiation by indication, outcome, and adherence in a realworld environment.…”

Integration of nurse-led virtual reviews with growth hormone device-linked patient adherence information: a mixed methods feasibility study

“…They have the capacity to provide support to clinicians through virtual reviews between clinic consultations and improve patient adherence to r-hGH treatment ( 15 ). While specialist nursing support to r-hGH treatment is common practice in the UK ( 16 ), supplemental nurse practitioner support to improve patient adherence and growth outcomes has not been assessed. We have proposed the logical integration of nurse-led virtual reviews (NVR) to easypod-connect™ adherence information in a prospective feasibility study.…”

Gene expression signatures predict response to therapy with growth hormone

“…Transcriptomic data have been used extensively in cancer tissues both to sub-type the tumour [ 14 – 16 ] and to predict response to therapies [ 17 , 18 ]. In contrast, in this study, we have used peripheral blood gene expression profiling as the source for gene expression profiles, and show that these patterns can be used to predict response to r-hGH in each year of treatment up to 5 years in two different growth disorders that account for ~60% of GH prescriptions in the USA, Europe and the UK [ 19 , 20 ].…”